[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT04360265":3,"trial-entities:NCT04360265":122,"trial-summary:NCT04360265":126},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":23,"study_type":26,"primary_purpose":27,"phases":28,"enrollment_info":30,"interventions":33,"primary_outcomes":45,"secondary_outcomes":51,"sex":65,"minimum_age":66,"maximum_age":66,"healthy_volunteers":67,"eligibility_criteria":68,"std_ages":76,"locations":80,"central_contacts":112,"overall_officials":113,"references":117,"see_also_links":118},"NCT04360265","LTFU-ABO-102","Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA","A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)","ENROLLING_BY_INVITATION","2027-08","2026-07","2026-07-17","2020-09-28","Ultragenyx Pharmaceutical Inc","INDUSTRY",true,"The main objective of this study is to evaluate the safety\u002Ftolerability and efficacy of UX111 (previously known as ABO-102) in participants with Mucopolysaccharidosis IIIA (MPS IIIA).","This is a multicenter, long-term follow-up study of patients with MPS IIIA who have participated in a prior clinical trial involving the administration of UX111 (NCT02716246 and NCT04088734). No investigational product will be provided. At approved sites adjuvant immunomodulatory (IM) therapy may be administered to selected participants. The Principal Investigator and\u002For caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive IM therapy.\n\nThis study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.",[19,20,21,22],"Mucopolysaccharidosis IIIA","MPS IIIA","Sanfilippo Syndrome","Sanfilippo A",[20,24,25],"Sanfilippo","Gene Therapy","INTERVENTIONAL","OTHER",[29],"PHASE3",{"count":31,"type":32},41,"ESTIMATED",[34,40],{"type":27,"name":35,"description":36,"armGroupLabels":37},"No Investigational Product","No investigational product will be administered in this follow-up trial.",[38,39],"Cohort A","Cohort B",{"type":41,"name":42,"description":43,"armGroupLabels":44},"DRUG","Adjuvant Immunomodulatory (IM) Therapy","The Principal Investigator and\u002For caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive IM therapy.",[38],[46,49],{"measure":47,"timeFrame":48},"Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)","Up to Year 5",{"measure":50,"timeFrame":48},"Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over Time",[52,55,57,59,61,63],{"measure":53,"timeFrame":54},"Cerebrospinal Fluid (CSF) Heparin Sulfate (HS) (Disaccharide) Exposure","Baseline, Up to Month 36",{"measure":56,"timeFrame":54},"Percent Change From Baseline in Prior Trial in CSF HS",{"measure":58,"timeFrame":48},"BSITD-III Receptive Communication Raw Score Over Time",{"measure":60,"timeFrame":48},"BSITD-III Expressive Communication Raw Score Over Time",{"measure":62,"timeFrame":54},"Annualized Percentage Change from Baseline in Prior Trial in Total Cortical Volume",{"measure":64,"timeFrame":48},"Survival","ALL",null,false,{"inclusion":69,"exclusion":72,"raw_text":75},[70,71],"Participants that have participated in a prior clinical trial in which they received UX111","Parent(s)\u002Flegal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule",[73,74],"Planned or current participation in another clinical trial that may confound the safety or efficacy evaluation of UX111 during this study","Any other situation or medical condition that precludes the participant from undergoing procedures required in this study","Inclusion Criteria:\n\n* Participants that have participated in a prior clinical trial in which they received UX111\n* Parent(s)\u002Flegal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule\n\nExclusion Criteria:\n\n* Planned or current participation in another clinical trial that may confound the safety or efficacy evaluation of UX111 during this study\n* Any other situation or medical condition that precludes the participant from undergoing procedures required in this study",[77,78,79],"CHILD","ADULT","OLDER_ADULT",[81,90,98,106],{"facility":82,"city":83,"state":84,"zip":85,"country":86,"geoPoint":87},"Nationwide Children's Hospital","Columbus","Ohio","43205","United States",{"lat":88,"lon":89},39.96118,-82.99879,{"facility":91,"city":92,"state":93,"country":94,"geoPoint":95},"Women's and Children's Hospital","North Adelaide","South Australia","Australia",{"lat":96,"lon":97},-34.90733,138.59141,{"facility":99,"city":100,"zip":101,"country":102,"geoPoint":103},"Vall d'Hebron Barcelona Campus","Barcelona","08035","Spain",{"lat":104,"lon":105},41.38879,2.15899,{"facility":107,"city":108,"country":102,"geoPoint":109},"Hospital Clínico Universitario de Santiago","Santiago de Compostela",{"lat":110,"lon":111},42.88052,-8.54569,[],[114],{"name":115,"affiliation":13,"role":116},"Medical Director","STUDY_DIRECTOR",[],[119],{"label":120,"url":121},"Ultragenyx Transparency Commitment","https:\u002F\u002Fwww.ultragenyx.com\u002Four-research\u002Four-clinical-trial-transparency-commitment\u002F",{"nct_id":4,"conditions":123,"biomarkers":125},[124],"Mucopolysaccharidosis Type IIIA",[],{"nct_id":4,"found":15,"summary":127,"prompt_version":137},{"design":128,"status":129,"heading":130,"summary":131,"follow_up":132,"word_count":133,"commitments":134,"compensation":135,"drugs_mentioned":136},"This is an interventional, multicenter, long-term follow-up study. It plans to enroll 41 participants.","completed","Follow-up Study for UX111 in MPS IIIA","This study is a long-term follow-up for individuals with Mucopolysaccharidosis IIIA (MPS IIIA), also known as Sanfilippo Syndrome Type A, who have previously received UX111 (formerly ABO-102) in an earlier clinical trial. The main goals are to continue checking the safety and how well UX111 works over time. No new UX111 will be given in this follow-up study. Some participants might receive additional immune-modulating (IM) therapy, decided by their doctor and caregiver. The study will track any side effects and changes in cognitive (thinking and learning) scores using the Bayley Scales of Infant and Toddler Development – Third Edition (BSITD-III) for up to five years. The study is currently unclear on its recruitment status and plans to enroll 41 participants.","Participants will be followed for up to five years to assess side effects and cognitive development.",120,"You would need to have participated in a prior clinical trial where you received UX111. You or your legal guardian must be willing to complete consent and follow study procedures and visit schedules.","Not stated in the trial record.",[],"v2"]